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PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency

ClinicalTrials.gov ID: NCT06302439

Public ClinicalTrials.gov record NCT06302439. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 5, 2026, 7:24 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Prospective Observational Patient Registry to Evaluate Disease Progression in Patients With ENPP1 Deficiency and Infantile-Onset ABCC6 Deficiency (GACI Type 2)

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette transporter protein subfamily C member 6 (ABCC6) Deficiency longitudinally. The registry will prospectively gather information about the genetic, biochemical, physiological, anatomic, radiographic, and functional manifestations (including patient reported outcomes \[PROs\]) of each disease during routine, standard-of-care visits, with the aim of developing a comprehensive understanding of the burden of illness and progressive nature of the disease.

Study identification

NCT ID
NCT06302439
Recruitment status
Active, not recruiting
Study type
Observational
Phase
Not listed
Lead sponsor
Inozyme Pharma
Industry
Enrollment
54 participants

Conditions and interventions

Eligibility (public fields only)

Age range
Not listed
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Jul 24, 2024
Primary completion
Aug 27, 2026
Completion
Aug 27, 2026
Last update posted
Aug 27, 2026

2024 – 2026

United States locations

U.S. sites
5
U.S. states
5
U.S. cities
5
Facility City State ZIP Site status
Ann and Robert H. Lurie Children's Hospital Chicago Illinois 60611
Boston Children's Hospital Boston Massachusetts 02115
Mayo Clinic Rochester Minnesota 55905
CLINILABS Drug Development Corp Eatontown New Jersey 07724
The Children's Hospital of Philadelphia (CHOP) Philadelphia Pennsylvania 19104

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 9 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT06302439, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Aug 27, 2026 · Synced Sep 5, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT06302439 live on ClinicalTrials.gov.

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